MUCOLIPIDOSIS TYPE IV

Clinical trials for MUCOLIPIDOSIS TYPE IV explained in plain language.

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Not yet finished but already full! 1 Terminated 1

First-in-Human gene therapy trial launches for devastating childhood disease

Disease control ENROLLING_BY_INVITATION

This is an early-stage study to see if a new gene therapy called AAV9.hMCOLN1co is safe for children with Mucolipidosis Type IV (MLIV), a rare and serious genetic disorder. The therapy aims to deliver a working copy of the faulty gene to try to control the disease. The study will…

Matched conditions: MUCOLIPIDOSIS TYPE IV

Phase: PHASE1 • Sponsor: The Children's Hospital of Zhejiang University School of Medicine • Aim: Disease control

Last updated Feb 27, 2026 15:19 UTC

MUCOLIPIDOSIS TYPE IV

Get alerted when new MUCOLIPIDOSIS TYPE IV trials appear

First-in-Human gene therapy trial launches for devastating childhood disease